Presymptomatic and symptomatic disease stage-associated outcomes of cerliponase alfa treatment in siblings with CLN2 disease

Brineura (cerliponase alfa) - BioMarin

https://onlinelibrary.wiley.com/doi/epdf/10.1002/jimd.70230

Lena Marie Westermann 1,2; Lara Yasmin Ruhberg 1; Susanne Lezius 5; Luca Hagenah 1; Eva Heemann 1; Miriam Nickel 1; Christoph Schwering 1; Christian Posern 1,2; Eva Tolosa 2,4; Angela Schulz 1,2

Jul 28, 2026